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Phase I/II Study of Total Body Irradiation, Cyclophosphamide, and Fludarabine Followed by Alternate Donor Hematopoietic Cell Transplantation in Patients With Fanconi's Anemia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00005898
Recruitment Status : Completed
First Posted : June 5, 2000
Last Update Posted : June 24, 2005
Information provided by:
Office of Rare Diseases (ORD)

Brief Summary:

OBJECTIVES: I. Determine the probability of engraftment with total body irradiation, cyclophosphamide, fludarabine, and anti-thymocyte globulin followed by HLA nongenotypically identical donor, T-cell depleted hematopoietic cell transplantation in patients with Fanconi's anemia.

II. Determine the incidence of acute and chronic graft-versus-host disease in these patients after undergoing this treatment regimen.

III. Determine the one-year survival rate in these patients after undergoing this treatment regimen.

IV. Determine the toxicity of this treatment regimen in these patients. V. Determine the incidence of relapse in patients with myelodysplastic syndrome or acute myeloid leukemia after undergoing this treatment regimen.

Condition or disease Intervention/treatment Phase
Fanconi's Anemia Drug: anti-thymocyte globulin Drug: cyclophosphamide Drug: cyclosporine Drug: filgrastim Drug: fludarabine Drug: methylprednisolone Phase 1 Phase 2

Detailed Description:

PROTOCOL OUTLINE: Donor bone marrow, peripheral blood, or umbilical cord blood is processed to harvest CD34+ cells.

Patients receive preparative cytoreductive therapy comprising total body irradiation on day -6; cyclophosphamide IV over 2 hours on days -5 to -2; fludarabine IV over 30 minutes on days -5 to -2; methylprednisone IV on days -5 to 24; anti-thymocyte globulin IV over 4-6 hours on days -5 to -1; cyclosporine IV over 2 hours every 12 hours (every 8 hours for patients less than 40 kg in weight) on days -3 to 180, and then tapering in the absence of graft-versus-host disease; hematopoietic cell transplantation on day 0; and filgrastim (G-CSF) IV starting on day 1 and continuing until blood counts recover.

Patients are followed at days 60, 90, and 180, and then annually for 3 years.

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Study Type : Interventional  (Clinical Trial)
Enrollment : 30 participants
Primary Purpose: Treatment
Study Start Date : February 2000

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 34 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


--Disease Characteristics--

  • Patients with a diagnosis of Fanconi's anemia AND Aplastic anemia OR Myelodysplastic syndrome OR Acute leukemia with or without chromosomal anomalies Aplastic anemia as defined by having at least one of the following: Platelet count less than 20,000/mm3 Absolute neutrophil count less than 500/mm3 Hemoglobin less than 8 g/dL
  • Myelodysplastic syndrome with multilineage dysplasia With or without chromosomal anomalies
  • Hematologic malignancy (e.g., acute myeloid leukemia, acute lymphoblastic leukemia, or B-cell non-Hodgkin's lymphoma)
  • No greater than 30% blasts in bone marrow or greater than 5% blasts in peripheral blood
  • No active CNS leukemia at time of transplantation
  • Must have an HLA A, B, DRB1 identical or 1 antigen mismatched related (nonsibling) or unrelated bone marrow, peripheral blood, or umbilical cord blood donor

--Prior/Concurrent Therapy--

  • Radiotherapy: No prior radiotherapy that would preclude total body irradiation
  • Surgery: Not specified

--Patient Characteristics--

  • Performance status: Karnofsky 70-100% OR Lansky 50-100%
  • Hematopoietic: See Disease Characteristics
  • Hepatic: No hepatic failure (e.g., coagulopathy or ascites)
  • Renal: Creatinine clearance at least 40 mL/min
  • Cardiovascular: Ejection fraction at least 45%
  • Other: No active uncontrolled infection within one week of transplantation No malignant solid tumor (e.g., squamous cell carcinoma of the head, neck, or cervix) within 2 years of transplantation Not pregnant or nursing Negative pregnancy test Fertile patients must use effective contraception

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00005898

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United States, Minnesota
University of Minnesota Cancer Center
Minneapolis, Minnesota, United States, 55455
Sponsors and Collaborators
University of Minnesota - Clinical and Translational Science Institute
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Study Chair: John E. Wagner, Jr. Masonic Cancer Center, University of Minnesota

Publications automatically indexed to this study by Identifier (NCT Number):
Layout table for additonal information Identifier: NCT00005898     History of Changes
Other Study ID Numbers: 199/15109
First Posted: June 5, 2000    Key Record Dates
Last Update Posted: June 24, 2005
Last Verified: July 2004

Additional relevant MeSH terms:
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Fanconi Anemia
Fanconi Syndrome
Hematologic Diseases
Anemia, Hypoplastic, Congenital
Anemia, Aplastic
Bone Marrow Diseases
Genetic Diseases, Inborn
DNA Repair-Deficiency Disorders
Metabolic Diseases
Renal Tubular Transport, Inborn Errors
Kidney Diseases
Urologic Diseases
Metabolism, Inborn Errors
Fludarabine phosphate
Antilymphocyte Serum
Methylprednisolone Hemisuccinate
Methylprednisolone Acetate
Prednisolone acetate
Prednisolone hemisuccinate
Prednisolone phosphate
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs